Opinion: California's Rare Disease Council Will Transform Care for People with ALS
For families affected by this rare, fatal disease, the journey is grueling — not only because of the diagnosis itself but due ...
Treatment to preserve sight in children with rare genetic disorder shows promise in clinical trial
A new treatment that could prevent blindness in children with the CLN2 type Batten disease has been trialed by clinicians and ...
FiercePharma3h
FDA rejects Intercept's ask for Ocaliva full nod in rare liver disease, but accelerated approval remains
In the wake of an unfavorable advisory committee meeting in September, Intercept Pharmaceuticals’ bid to win full approval ...
US FDA declines full approval for Intercept's liver disease drug
The U.S. Food and Drug Administration on Tuesday declined full approval for Intercept Pharmaceuticals' liver disease drug, ...
STAT8d
My son’s rare disease was renamed ASMD. Why are people still calling it Niemann-Pick?
Eponyms — diseases named after a person — can cause serious confusion. That is particularly true in one rare disease ...
New treatment helps children with rare spinal condition regain ability to walk
Researchers at Kennedy Krieger Institute's International Center for Spinal Cord Injury (ICSCI) have made a remarkable ...
Cherokee Nation Hosts First Rare Disease Summit to Address Overlooked Health Care Needs
Cherokees deserve help, treatment and hope – especially those who’ve long felt unseen by the health care system due to their ...
EurekAlert!1d
Stand Up to Cancer collaborates with Johnson & Johnson to explore targeted therapies for rare disease linked to blood cancers
Stand Up To Cancer® (SU2C) today announced a collaboration with Johnson & Johnson aimed at supporting research testing a ...
Targeted Oncology22h
Elraglusib Gains FDA Rare Pediatric Disease Designation in Ewing Sarcoma
Elraglusib has been granted rare pediatric disease designation from the FDA for the treatment of patients with Ewing sarcoma.
Science News4d
A common drug may help treat a rare genetic disease
Ibuprofen counters problems caused by mutations in the MAN1B1 gene, fruit fly tests show. Early results in three children are ”fairly positive.” ...
FiercePharma7d
Arrowhead targets rare disease patients, pushing message that hope is on the horizon
Arrowhead Pharmaceuticals has begun laying the groundwork for its entry into a rare disease market, kicking off an awareness ...
The American Journal of Managed Care4d
Rare Disease
Patient input and experiences play a crucial role in advancing rare disease research and therapy development, as they help define the disease, inform clinical trial design, and influence ...